Stem Cell Stocks: 10 Biggest NASDAQ Companies

Market cap: US$207.4 billion

First on this list of the top NASDAQ stem cell stocks is multinational pharma and biotech firm AstraZeneca, which also specializes in other therapeutic areas, including oncology, cardiovascular diseases, the respiratory and central nervous systems and pain control.

While it may be the largest company on this list by market cap, the pharma giant has been slower than its peers in staking a position in the cell therapy market. Its long-term strategy in this area is to advance "off-the-shelf" allogenic CAR-T therapies that directly target cancer cells. The company is also looking at stem cell therapies to treat life-threatening chronic kidney disease and liver disease.

Market cap: US$153.28 billion

A global leader in biotech, Amgen is heavily invested in gene-based research and uses advanced human genetics to develop and manufacture therapeutics targeting a variety of diseases with unmet medical needs. Amgen states on its website that its goal is to take an industrial-scale approach to gene-based research.

The biotech firm has a multi-year fund agreement with Canada's Center for Commercialization of Regenerative Medicine (CCRM), which specializes in developing and commercializing regenerative medicine-based technologies and cell and gene therapies. Under the multi-year agreement, both CCRM and Amgen will fund early stage regenerative medicine-based technologies and therapies with research conducted in institutions that are part of CCRM’s global network.

Market cap: US$137.9 billion

French multinational pharmaceutical company Sanofi develops therapeutic products for diabetes and cardiovascular diseases, oncology, immunology, multiple sclerosis, rare diseases, rare blood disorders and vaccines. Its product portfolio includes Mozobil, a hematopoietic stem cell mobilizer that helps bone marrow release stem cells into the bloodstream for transplant into the body.

In 2021, the firm bolstered its regenerative medicine business through the US$1.9 billion acquisition of Kadmon, adding FDA-approved stem cell transplant product Rezurock to its portfolio. Sanofi has since partnered with privately held biotech company Scribe Therapeutics to develop CRISPR-based cell therapies targeting cancers.

Market cap: US$97.94 billion

Global biopharmaceutical company Gilead Sciences specializes in developing breakthrough medicines to prevent and treat serious diseases such as HIV, viral hepatitis and cancer.

One of the stem cell therapies in its product portfolio is Yescarta, a CAR-T cell therapy for blood cancer and the first such therapy for certain types of non-Hodgkin's lymphoma. Yescarta aids a patient’s immune system in fighting the disease. Gilead currently has at least eight cell therapy candidates in its product pipeline, including two in Phase 3 clinical studies targeting high-risk lymphomas.

Market cap: US$96.26 billion

Vertex Pharmaceuticals has developed a number of approved treatments for cystic fibrosis, and has a pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes.

The global biotech firm further expanded its regenerative medicine portfolio with the US$320 million acquisition of ViaCyte and its stem cell-based type 1 diabetes treatment VCTX210, which is currently in Phase 1/2 clinical trials. VCTX210 is an investigational, gene-edited, immune-evasive, allogeneic stem cell therapy that Vertex is developing in collaboration with CRISPR Therapeutics.

Vertex and its partner CRISPR Therapeutics have received validations from the European Medicines Agency and from UK authorities for filings concerning product candidate exagamglogene autotemcel, formerly known as CTX001, as a one-time treatment for severe sickle cell disease and transfusion-dependent beta thalassemia. The drug candidate is set to become the first ever CRISPR-based treatment approved for the US market in early 2024.

Market cap: US$35.23 billion

Biotechnology and pharmaceutical company Moderna is a pioneer in the field of mRNA. The company’s assets include a diverse clinical portfolio of vaccines and therapeutics, plus a large intellectual property portfolio in areas such as mRNA and lipid nanoparticle formulation; it also has an integrated manufacturing plant that allows for both clinical and commercial production.

These assets, along with Moderna’s network of domestic and overseas government and commercial collaborators, allowed for the rapid development of one of the world’s most effective COVID-19 vaccines.

The other therapeutics and vaccines in the company’s pipeline are targeting infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and autoimmune diseases. Moderna has 23 development programs underway across these areas.

Market cap: US$23.37 billion

Immunotherapy company BioNTech is advancing novel therapies for diseases such as cancer. The company combines computational discovery and therapeutic drug platforms to rapidly develop new biopharmaceutical products. BioNTech’s portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, CAR-T cell therapies, bispecific checkpoint immuno-modulators, targeted cancer antibodies and small molecules.

Besides its diverse oncology pipeline, the company is best known today for its mRNA vaccine development and in-house manufacturing capabilities. In addition to its COVID-19 vaccine, created along with partner Pfizer (NYSE:PFE), BioNTech is engaged in collaborative partnerships aimed at assembling mRNA vaccine candidates for a range of infectious diseases.

Market cap: US$19.19 billion

Biotechnology company BeiGene specializes in the development of drugs for cancer treatment across a broad range of areas, including esophageal squamous cell carcinoma, non-small cell lung cancer, mantle cell lymphoma, non-Hodgkin's lymphoma and ovarian cancer. Its clinical development pipeline includes 12 advanced Phase 3 programs.

BeiGene has a strategic partnership with Shoreline Biosciences for the development and commercialization of a genetically engineered natural killer cell therapy. “Cell therapy inevitably will become another pillar of therapeutics in the future, especially for oncology,” Alex Huang, BeiGene's vice president and head of cell therapy, told GeneOnline.

BeiGene's bruton tyrosine kinase inhibitor Brukinsa gained FDA approval in early 2023 for patients with chronic lymphocytic leukemia or small lymphocytic leukemia, both forms of non-Hodgkin's lymphoma.

Market cap: US$15.96 billion

BioMarin Pharmaceutical develops and commercializes therapies for patients with serious life-threatening genetic diseases and medical conditions. BioMarin’s head of research and early development, Dr. Kevin Eggan, is a pioneer in stem cell biology.

The global biotech company focuses on diseases with critical unmet medical needs, as well as products with an opportunity to be first to market or to offer significant benefits over existing products. BioMarin's portfolio includes eight commercial products and multiple clinical and preclinical product candidates across therapeutic areas.

Market cap: US$3.51 billion

A leader in gene editing, CRISPR Therapeutics uses its proprietary CRISPR/Cas9 platform to develop transformative gene-based medicines for serious diseases. The company says its gene-editing technology “allows for precise, directed changes to genomic DNA." CRISPR Therapeutics’ therapeutic programs target hemoglobinopathies, oncology, regenerative medicine and rare diseases.

Along with its partner Vertex Pharmaceuticals, the company currently has a stem cell therapy for the treatment of type 1 diabetes in clinical trials. The treatment applies CRISPR Therapeutics’ gene-editing technology to Vertex's proprietary stem cell capabilities for the generation of pancreatic cells designed to evade recognition by the immune system.